A groundbreaking experimental gene therapy has offered significant improvements in vision for four toddlers suffering from a rare genetic form of blindness. Prior to treatment, these children were legally blind, only capable of identifying light versus dark. Post-therapy, parents observed transformative changes, with some toddlers beginning to draw and write. The treatment involves injecting healthy genes into the eye and builds on prior successes with similar therapies. One case featured Jace, whose parents tirelessly sought a diagnosis, ultimately finding hope through participation in the London trial, spotlighting the importance of early intervention in such cases.
The rare genetic condition means the babies' vision deteriorated very rapidly from birth. Before therapy, they were legally blind, able only to distinguish light.
Jace's parents noticed something wasn't right about his eyesight at eight weeks old, when he didn't smile or look at them like other babies.
After gene therapy, parents reported improvements in their children's vision, with some able to start drawing and writing, marking significant life changes.
The gene therapy involves injecting healthy copies of a defective gene into the eye as a promising early intervention for severe genetic blindness.
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